Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-
associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential …

Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years
ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species …

Adeno-associated virus (AAV) vectors have emerged as a gene-delivery platform with
demonstrated safety and efficacy in a handful of clinical trials for monogenic disorders …

Gene therapy has a history of controversy. Encouraging results are starting to emerge from
the clinic, but questions are still being asked about the safety of this new molecular …

Parvoviruses, infecting vertebrates and invertebrates, are a family of single-stranded DNA
viruses with small, non-enveloped capsids with T= 1 icosahedral symmetry. A quarter of a …

Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …

Adeno-associated virus (AAV) serotypes differ broadly in transduction efficacies and tissue
tropisms and thus hold enormous potential as vectors for human gene therapy. In reality …

The structure of the adeno-associated virus (AAV-2) has been determined to 3-Å resolution
by x-ray crystallography. AAV is being developed as a vector for gene therapy to treat …